Lessons From Effective Value-Based Care Models in Hematopoietic Stem Cell Transplantation

Abstract: Patient-centered medical homes (PCMHs) have been designed to offer coordinated care delivery across the entire spectrum of care, aiming to provide optimal care for patients given their individual circumstances. The hope is that better care coordination will reduce cost and resource utilization and lead to better cost value for health care. Clinical pathways and guidelines are tools that can measure and improve care, where outcomes can be measured against prior historical data. Most of the models for PCMHs are primary care models. The hematopoietic cellular therapy (HCT) community has offered such a care model for high-acuity complex patients for several decades despite never having the label of “medical home.” These patients have been subject to bundled payments and have had outcomes reported in a national outcomes database, because these programs started as clinical research programs with treatment delivered according to research protocols. More recently, these protocols for routine care have morphed into clinical pathways and guidelines. Measurements can be compared with a center’s historical outcomes as these HCT centers are required to track outcomes of patients in an institutional database. A center’s outcomes can also be compared to international registry outcomes for which centers are federally mandated to report their allogeneic transplant outcomes. The experiences of HCT centers that deliver this type of coordinated, medical home care provide useful insight for care settings moving toward a value-based care model.

Complex clinical care is often best administered with coordinated integration of inpatient and outpatient services^1-6 Management of specific problems can be done by consulting clinical guidelines, where the guidelines can be assessed for utility and validity with observation of results compared with prior historical outcomes. But patients rarely present with a solitary problem; rather, they have a problem requiring assessment, intervention, and treatment in the context of multiple comorbidities, which are not always fully assessed and adequately treated. Often treatment of one disease will affect treatment of other comorbidities, sometimes adversely. 

Not all patients requiring complex care have fully functional support systems enabling optimal care delivery in the lowest cost venue. Patient-centered medical homes (PCMHs) emphasize a coordinated care model to manage circumstances unique to that patient’s medical and social circumstances.^7,8 However, coordinated care may not reduce costs as it tries to provide optimal care.^9,10 For this reason, a tool to reduce costs and reduce variation should be used as well, such as clinical pathways. 

The area of hematopoietic cellular therapy (HCT) provides an example of how complex clinical care can be coordinated and managed in a medical home model and by using clinical pathways.¹¹ HCT is a complex therapy used for the treatment of bone-marrow-derived cancers like leukemia, lymphoma, myeloma, bone marrow failure diseases like aplastic anemia and inherited metabolic, and immunologic disorders. This therapy shifted from being a solely investigational research therapy to a standard-of-care option for many patients. Because it was a research therapy originally, care was determined and executed according to institutional review board-approved clinical research protocols. As the therapy matured and became standard of care, much of the care for common complications and preventive therapy was determined by clinical pathways and guidelines. 

Those in the HCT field have and continue to track and compare outcomes in an effort to improve survival and reduce mortality. In 1972, HCT-associated organizations established an international outcomes database titled the International Bone Marrow Transplant Registry (IBMTR), which, after a merger with the National Marrow Donor Program, is now part of the Center for International Blood and Marrow Transplantation Research (CIBMTR).¹² The CIBMTR was awarded the contract to run the Stem Cell Transplant Outcomes Database, where all US allogeneic HCT outcomes are tracked and reported as required by the 2005 Stem Cell Therapeutic and Research Act.¹³ Since its inception in 1972, the IBMTR and its successor CIBMTR has facilitated over 1000 peer-reviewed publications with dramatic impacts on changes in standard of care, resulting in consistent improvement in transplant survival over the past 40 years. With the use and assessment of guidelines, care measurement, a comprehensive outcomes registry (CIBMTR), and its placement in academic medical centers, HCT care delivery provides a model for exploring options for value-based care in settings outside of primary care and relevant to complex specialty care. ^14,15

Hematopoietic Cellular Therapy Background

Originally, HCT was to provide hematopoietic stem cell rescue after treatment with high doses of chemotherapy and radiation therapy where the principle toxicity of such therapy was bone marrow failure. Options for rescue graft were either from the patient, being harvested prior to the high-dose therapy as an autologous donor, or the graft came from another person (allogeneic donor) closely immunologically matched with the intended recipient patient. Further science demonstrated that the graft from an allogeneic donor had an immunologic “graft vs tumor” effect. Even an autologous transplant was associated with regrowth of the immune system that allowed for added benefit. 

HCT is associated often with a month-long initial hospital stay as the high-dose therapy is administered, followed by the graft infusion, and a pancytopenia phase while awaiting the graft recovery. HCT patients are at high risk of multiple complications including severe infection, debilitation, inability to adequately hydrate via oral intake, electrolyte loss, immunologic complications, and recurrent peripheral blood cytopenias—all of which may result in high rates of hospital readmission.^16,17

Patients who undergo HCT have frequent outpatient therapy visits after initial transplant discharge, often coming in 3 to 7 times per week for a prolonged period over several months. These visits require immediate routine laboratory testing with quick turnaround of results, intravenous (IV) hydration, IV electrolyte replacement, IV antibiotics, and urgent transfusion of blood products. Blood products for these severely immunocompromised patients must be irradiated and often are difficult to crossmatch given that HCT donor and recipient are not always of the same blood type.^18-20

HCT patients are much sicker and more complex than almost any other type of oncology patient. The only comparable group in terms of acuity is patients with acute leukemia, who will often proceed to hematopoietic cell transplant for treatment.^21,22 Consequently, acute leukemia patients are frequently managed by the same team and care centers as patients undergoing HCT.

HCT Care Delivery

Because of the high acuity of this patient population, HCT care typically has been provided as organized, integrated inpatient and outpatient team-based care. HCT patient care is provided by the same physician and advance practice professional providers in both inpatient and outpatient settings. Hospitalists are not utilized for these patients. Inpatient teams usually include one HCT physician, 2 to 4 advance practice professionals, and a clinical pharmacy specialist with a specialized transplantation background. Inpatient care by HCT physician providers and advance practice professionals is usually done on a rotational basis, with rotations lasting an interval ranging from 1 week to 1 month.

Outpatient care in most, but not all, centers has one HCT physician provider designated as the primary doctor in charge of an individual patient. This physician provider will see the patient frequently and be involved with or make most major treatment planning decisions. Urgent care or management of the patient on clinical pathways is often guided by the advance practice professionals and nurses with specialized background training in HCT. Both inpatient and outpatient HCT care teams also include nurse educators, clinical nurses, social workers, nutritionists, physical therapists, occupational therapists, and speech therapists with specialized HCT specific training. Physician specialties often have physician providers well-versed in and clinically focused on HCT-related complications. 

Payment Models for HCT Care

Medicare pays for transplant services differently depending on whether the patient is enrolled in traditional fee-for-service Medicare or enrolled in a Medicare Advantage plan. For traditional fee-for-service Medicare, Medicare pays for initial HCT hospitalization that includes cell infusion with special HCT-specific Medicare Severity-Diagnosis Related Groups (MS DRGs): MS DRG 14 for allogeneic HCT, MS DRG 16 for autologous HCT with comorbidities, and MS DRG 17 for autologous HCT without comorbidities.²³ For readmissions, traditional Medicare fee-for-service is problematic, as those readmissions have no specialized, HCT-specific DRGs. They will utilize DRGs whose payment is based on usage in a less complex community population, such as sepsis and fever. This results in an underpayment for services for more complicated HCT readmissions post-transplant. For the latter scenario, the Medicare Advantage plan contracts with a HCT center, usually the same contract as utilized with the commercially insured population.

Commercial payers have paid for HCT care using modified case rates or bundled payments since the early 1990s. These case rates often pay for components of care or phases. These phases include initial evaluation, donor search, pre-HCT assessment, donor cell collection, initial HCT care with preparative chemotherapy and cell infusion, and post-HCT care. ^22,24 Professional services are often but not always bundled into the bundled payment. The professional services total charges usually represent less than 10% of the total bill. The procedural services utilized by HCT are few and have low relative value units. The HCT physician services are largely evaluation and management services, reflecting the need to manage complex multiorgan dysfunctional patients.

Both initial HCT care and post-HCT care require unpredictable amounts of resource consumption; thus, case rates with commercial payers always have outlier clauses for catastrophic cases. Pre-HCT comorbidities cannot be easily excluded from case rates due to administrative issues processing claims, and care for these diseases often cause unpredictability. Additionally, severe HCT complications occur at 30% to 50% incidence and cannot often be prevented.²² Such complications, if they occur, can cause a catastrophic resource consumption. With such a high proportion of care having extreme variance, HCT centers have been reluctant to have fixed bundles with commercial payers without outlier clauses. As Medicare Access and CHIP Reauthorization Act of 2015 (MACRA) pushes the United States toward value-based payment, physicians and hospitals will be measured on costs per patient utilized for treating certain episodes of care in the Merit-based Incentive Payment System (MIPS) portion of MACRA. The alternative payment model (APM) portion of MACRA will likely also look at the same MIPS episodes.

An essential need when paying for value is acuity adjustment in coding and claims data. Acuity adjustment has never been adequately addressed, and physicians have never, until this statute passed, been incentivized to comprehensively code for all comorbidities with acuity adjusters. For example, HCT patients often get diabetes, but that diabetes is secondary to prolonged usage of corticosteroids to treat HCT complications. Diabetes secondary to corticosteroids poorly responds to all hypoglycemic agents including insulin. So, in addition to coding diabetes, the physicians will need to code for steroid complications. For chronic obstructive pulmonary disease (COPD), physicians will need to code when patients are CO₂ retainers, as those COPD patients do worse.

Psychosocial factors and social determinants also play a role in outcome cost measures for episodes of care. For example, patient noncompliance with care is rarely coded for claims data. Causes of noncompliance, like inadequate insurance, family dynamics problems, and abusive behavior, are also not coded. Cost measures will likely necessitate physicians coding these factors. The Centers for Medicare & Medicaid Services (CMS) does not recognize many of the psychosocial factors such as can patients afford the medications, how rural is the home setting, and is the home so rural there are no home care agencies. As an example of the relevancy, I personally have patients in Oregon who live 45 minutes even from a gas station; there are no agencies to help them transition to home care.

What HCT has been concerned with regarding family support systems will now be shared as a concern by other providers in PCMHs. Because none of this data is in claims, these drivers of cost and resource consumption have never been historically tracked. Only diagnostic data found on claims could be tracked. Rules for what is allowable in claims data comes from what CMS is allowed to pay for or to adjust payment for.

A proposed value-based care model was presented at the Physician-Focused Payment Model Technical Advisory Committee (PTAC) by Hackensack Hospital in New Jersey.²⁵ This bundle focused on outpatient therapy for breast and lung cancer. This model proposed a bundled payment for outpatient supportive labs and IV hydration for breast cancer and lung cancer but excluded chemotherapy drugs; this model is unlikely to be applied to HCT and acute leukemia as the latter have much greater resource consumption than breast and lung cancer chemotherapy, which do not cause the severity of pancytopenia. The difference is that these cancers rarely infiltrate the bone marrow, hence there is less cytopenia. Even this model excludes chemotherapy, as that is dependent on cancer stage, cancer molecular phenotyping, and prior response plus patient desire for therapy.

Clinical Pathways and HCT Care Delivery

With the complexity of HCT care delivery, HCT care has remained in academic centers and has not devolved to community practices despite being delivered for over 40 years. It is a rare exception of an older therapy not leaving academic medical centers. Academic physicians are expected to publish peer-reviewed literature or leave the academic center—the crude vernacular phrase is “publish or perish.” This resulted in desire on most HCT physician providers to treat patients on a clinical research trial and have the outcome of that trial published. Because historically most patients were treated in clinical trials, it was natural for HCT patients to be managed on clinical pathways, particularly for some of the common HCT supportive care problems like neutropenic fevers, electrolyte replacement, and transfusion usage. Issues that often affect HCT outcomes like preparative regimens prior to HCT infusion and immunosuppression for pre-emption of graft-versus-host disease have more frequently been a source of enrollment of HCT patients in clinical trials. As the clinical trials in this area have matured, the clinical trials evolve at times into clinical pathways. The HCT patients often have rare and unpredictable toxicities of transplant where there are insufficient numbers to do a proper clinical trial, so these are often managed via pathways to efficiently assess care. HCT patients often require expensive drugs, and given the case rate payment, clinical pathways have been utilized to control usage of these expensive drugs and evaluate their clinical efficacy.

The HCT arena has struggled with pathways for integrative management of multiple comorbidities with which patients often begin HCT. Because HCT is often the only curative therapy for a patient, these patients cannot be denied access to care. However, how to have pathways for patients that integrate heart failure, hypertension, coronary artery disease, and diabetes along with HCT care has been a challenge. There is too much heterogeneity of comorbidities to have a uniform strategy. HCT is not unique here, as this comorbidity management strategy is a problem for all specialties. This is even more of a problem for clinical trial eligibility, as clinical trials assume the patient has only one medical problem, and the trial measures the outcome response without confounders. In reality, most patients, and particularly the Medicare beneficiaries, have multiple medical problems. This is a struggle for clinical pathways as well and will be a struggle for PCMHs that will be treating patients on treatment algorithms. They will struggle in balancing treatments with 2 or more diseases where treatment for 1 disease adversely impacts treatment for another disease.

Social situation and caregiver support play significant roles in HCT care.^{11,14, 22} While the intensity and complexity of HCT care makes these items more critical, these social determinants are a problem for others trying to deliver treatments according to clinical pathways and algorithms. HCT requires patients to have caregivers and family supportive systems, but not everybody has caregivers that are equally competent. Because the denial of HCT therapy often means certain, imminent death of the patient, HCT centers frequently proceed even when a family caregiving support system is less than adequate. 

Additional PCMH Considerations 

As HCT care delivery systems have rarely evolved beyond the academic medical centers, few community hospitals have the facilities or physician practices to support these patients for the first 6 months to 1-year post-HCT. The medical oncology physician workforce is not keeping up with retirements,^11,26 as demand for services are growing with both cancer prevalence being higher in an aging population and the explosion of new cancer treatments that is lengthening survival and converting more cancers into chronic diseases.²⁷

Medical oncology practice is focused on new outpatient treatments for solid tumors rather than management of complex multi-organ dysfunction that are common with HCT patients. Revenue stream for medical oncology practices is primarily based on chemotherapy treatments rather than support services like fluid management, transfusion, electrolyte replacement, or antibiotic therapy. Solid tumor medical oncology has moved almost entirely to outpatient therapy whereas HCT patients, for the first year, have frequent readmissions and intensive, almost daily outpatient therapy.^{11,14,18,21,22} HCT centers had limitations on beds, so they also had to expand outpatient services. The bundled payments from commercial payers reflected this integrated inpatient and outpatient care delivery. However, this complex care delivery was only doable in venues where practices and hospitals were fully integrated like academic medical centers. HCT bundled payments required outlier clauses for centers to assume partial financial risk given heterogeneity due to the disease presentation of HCT and also due to the uncertainty given lack of uniformity of costs associated with comorbidities and needed caregiver support.

Conclusion

While the medical home model helps to provide integrated care in a complex medical milieu, the medical home lacks the ability to control the financial toxicity risk given inability to control comorbidities, patient social and caregiver support, and the uncertainty of resource consumption in the HCT procedure. HCT treatment centers illustrate well that complex clinical care is best administered with coordinated integration of inpatient and outpatient services. Management of specific problems can be done by clinical guidelines where the guidelines can be assessed for utility and validity with observation of results compared with prior historical outcomes. PCMHs will likely emphasize coordinated care to manage circumstances unique to that patient’s medical and social circumstances. HCT centers with coordinated care have not demonstrated reduced costs as patient comorbidities drive cost as much as the major disease processes. Medical homes for complex patients that rely on pathways and guidelines will provide tools for measurement of care and thus hopefully improve outcomes.

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